tbuckley/cabot-search huggingface dataset:
PB2086: SIMULTANEOUS DIAGNOSIS OF MULTIPLE MYELOMA AND MYELODYSPLASIA: CASE REPORT.
Authors: Khaled Benabed, Janine Ferey, Manon Riviere, Cheze Stephane, Mohamed Yacine Benabed
Journal: HemaSphere (2023)

Topic: 13. Myeloma and other monoclonal gammopathies - Biology & Translational Research Background: We report here the case of an 82-year-old patient, Caucasian, with no notable history, presenting a long-standing thrombocytopenia (107 G/L) and a low-level IgA kappa monoclonal gammopathy. CLINICALLY, the patient only complained of fatigue. We note the existence of trophic lesions of the distal extremities with chilblains (fingers and toes) without evidence for an underlying autoimmune disease. A first bone marrow aspiration shows some signs of DYSGRANULOPOIESIS, DYSERYTHROPOIESIS and 8% of DYSTROPHIC PLASMA CELLS. IMAGING (whole-body MRI) does not show suspicious bone lesions, except for a degenerative spine. CGH array analysis and FISH on sorted plasma cells, objectified recurrent abnormalities in the Multiple Myeloma (MM): a 1q21 gain; a gain of chromosomes 9 and 19 and a loss of chromosome 13; deletion of the long arms of a chromosome 14. No deletion of TP53 was demonstrated. Ten months later, because of the progression of thrombocytopenia and the occurrence of normochromic macrocytic non regenerative anemia and neutropenia, a new bone marrow aspiration is performed, this time finding a 20% dystrophic plasma cell infiltration associated with MDS with dysplasia concerning the 3 lineages, without excess blasts. CONVENTIONAL KARYOTYPING, out of 21 mitosis, returns to normal. The serum free light chains kappa/lambda ratio is 2.207. The Next Generation Sequencing (NGS) Myeloid Panel study detects two ASXL1 mutations, providing a clonality argument for a myeloid hemopathy, in this case, Myelodysplasia (MDS) (1). The patient does not respond to treatment with recombinant erythropoietin (EPO) combined with short-time corticosteroid therapy for one week. THE EVOLUTION was marked by the occurrence in October 2022 of a febrile delirium. The complete blood count now shows very dystrophic circulating plasma cells (approximately 165/mm3). Analysis of Cerebrospinal Fluid (CSF) shows the presence of Listeria monocytogenes, and cytologically a contingent of atypical plasma cells. Recently, the patient was hospitalized again for an ischemic stroke (3). Aims:Methods: Results:Summary/Conclusion: The simultaneous diagnosis of MM and MDS without a history of chemotherapy and/or radiotherapy is rarely reported in the literature. Further research is needed to elucidate the underlying mechanisms predisposing to a coexistence of MM and MDS. The contribution of new generation diagnostic tools have now become essential for difficult and complex cases as illustrated in that case. Other similar cases and additional studies are necessary to establish therapeutic recommendations which still remain uncodified in this type of case. Keywords: Multiple myeloma, Thrombosis, ASXL1, Myelodysplasia
PB2007: INTEGRATION OF TELEHEALTH IN DAILY CLINICAL PRACTICE IN PATIENTS WITH MYELODYSPLASTIC NEOPLASMS - STATUS QUO AND FUTURE PERSPECTIVES
Authors: Reinhard Stauder, Adrian Lühring, Stefanie Tipelius, Gerhard Rumpold, Patrick Künstner
Journal: HemaSphere (2023)

Topic: 10. Myelodysplastic syndromes - Clinical Background: Electronic assessment of patient reported outcomes (ePROs) and of patients´ activities offers new perspectives in the individualized clinical care in patients with Myelodysplastic Neoplasms (MDS). Aims: To report experience from a single institution on the implementation of ePROs in daily practice and to discuss these results in context with the literature. Methods: Patients with MDS from our outpatient clinic were screened from June 2021 until November 2022. Recording of health-related quality of life (HRQoL) was performed using the electronic scores EORTC QLQ-C30, EORTC QLQ-FA12 and EuroQol-5D-5L in German language. The software Computer-based Health Evaluation System (CHES) was provided by ESD. This study was approved by the local ethics committee and written informed consent was obtained from all participating patients. Results: Out of 34 consecutive patients finally 17 individuals (median age 71 years (range 39 – 84 years; male 59%) were included. Patients were in the IPSS-R very low/low (n=11), intermediate (4) and high-risk (1) category and one patient suffered from a CMML (HP-subtype). Personal reasons (47%), restricted technical skills (29%), impaired cognitive capacities (12%) and not being native in German language (12%) were the main reasons for non-inclusion in this study. Six patients completed the scoring independently and remotely. Another six patients were supported by giving them access to the questionnaire at our institution. Five patients needed help from a study nurse who read the questions and filled in the answers. Overall, consecutive assessments were feasible with a median of five measurements (range 1-28). Based on electronic EORTC QLQ-C30 scoring, impairments were reported in physical and emotional function (65%), in role (35%) and in cognitive function (29%). Fatigue (71%), pain (65%), dyspnea (65%) and insomnia (47%) were the most prevalent symptoms. Likewise, MDS-patients complained about interference with daily life (65%), physical and emotional fatigue (53% and 47% respectively), social sequelae (41%) and cognitive fatigue (24%) as assessed by an electronic EORTC QOL-Fatigue score. Slight/moderate problems were reported in the EQ-5D-5L dimensions pain (53%), mobility (41%), usual activities (29%), anxiety (29%) and self-care (6%). Severe/extreme problems were reported in usual activities (12%), self-care (6%) and pain (6%). EQ-5D VAS was 69 (median) (range 30-98). Summary/Conclusion: This pilot study reports on the successful integration of ePROs in MDS-patients. The impact of these findings and our ongoing project on the evaluation of patients´physical activities by smart devices will be discussed in the context of the literature and future clinical studies. Keywords: MDS, Quality of life, Patient reported outcomes
PB2136: THE VALUE OF PERIPHERAL BLOOD LYMPHOCYTE SUBSETS IN PREDICTING THE PROGNOSIS OF NEWLY DIAGNOSED MULTIPLE MYELOMA
Authors: Zhaoyun Liu, Xianghong Zhao, Rong Fu
Journal: HemaSphere (2023)

Topic: 14. Myeloma and other monoclonal gammopathies - Clinical Background: Multiple myeloma(MM), which is characterized by the proliferation of malignant plasma cells producing monoclonal immunoglobulins, is the second most common hematological malignancy. With deep understanding in MM, it is stressed that the role of immune dysregulation in the pathogenesis of MM. The certain immune profiling has value in predicting the prognosis of MM patients. Aims: To explore the value of peripheral blood lymphocyte subsets in predicting prognosis in newly diagnosed multiple myeloma (NDMM) patients. Methods: 110 patients in Tianjin Medical University General Hospital were included. The percentage and absolute numbers of CD3+ T cells, CD3+CD4+ T cells, CD3+CD8+ T cells, CD3-CD56+ NK cells and CD3-CD19+ B cells were detected. The least absolute shrinkage and selection operator (LASSO) regression was conducted to investigate the independent risk factors. Best cut-off values were calculated by ROC analysis. Univariate and Multivariate COX analysis was used to evaluate the independent prognostic factors of overall survival. Kaplan-Meier curves and log rank test were used for analyzing overall survival. Results: We selected the predicting factors by using LASSO regression. The risk scores was calculated by the corresponding lambda values(the percentage of CD3+ T cells: -0.02670097, CD3+CD4+ T cells: 0.03081192, CD3-CD56+ NK cells: -0.02376163 and the absolute numbers of CD3+CD4+ T cells: -0.001719961, CD3-CD56+ NK cells: -0.00006968683. The best cut-off value is -2.022(AUC=0.750). We divided the patients into two groups(high risk: 37 vs low risk 73). The serum calcium(P<0.001) and the creatinine(P=0.001) were different in the patients’ characteristics between two groups). The univariate and multivariate analysis revealed that the values≤-2.022 was an independent prognostic factor for predicting OS(P<0.001).The 3-year OS rate of patients was higher in low risk group than high risk group(91.8% vs 56.8%, P<0.001). We further analyzed the significance in predicting prognosis of values in different staging system for MM. The 3-year OS rate is different in both low-medium and high risk in different staging system (ISS I-II:92.5% vs 61.5%,P=0.002; ISS III: 90.9% vs 54.2%, P=0.001; R-ISS I-II: 93.2% vs 58.3%, P<0.001; R-ISS III: 85.7% vs 46.2%, P=0.02; R2-ISS I-II: 100% vs 66.7%, P=0.001; R2-ISS III-IV: 87.2% vs 54.8%, P<0.001). Summary/Conclusion: The findings suggested that lymphoid subsets have prognostic value in NDMM patients and mightbe a potential factor in distinguishing patients at different risk.Keywords: Multiple myeloma, Immunity
PB2104: SOLUBLE B-CELL MATURATION ANTIGEN AS A MONITORING MARKER FOR MULTIPLE MYELOMA
Authors: Ádám Wiedemann, Virag Reka Szita, Róbert Horváth, Attila Sebő, András Dávid Tóth, Szabolcs Svorenj, Gergely Szombath, Tamas Masszi, Gergely Varga
Journal: HemaSphere (2023)

Topic: 14. Myeloma and other monoclonal gammopathies - Clinical Background: Response to treatment in multiple myeloma (MM) is routinely measured by serum and urine M-protein and free light chain (FLC), as described by the International Myeloma Working Group (IMWG) consensus statement. A non-negligible subgroup of patients however present without measurable biomarkers, others become oligo- or non-secretory during recurrent relapses. Aims: The aim of our research was to evaluate soluble B-cell maturation antigen (sBCMA) as a monitoring marker measured concurrently with the standard monitoring in MM patients at diagnosis, at relapse and during follow up, in order to establish its potential usefulness in oligo- and non-secretory disease. Methods: sBCMA levels were measured in 149 patients treated for plasma cell dyscrasia (3 monoclonal gammopathy of unknown significance, 5 smouldering MM, 7 plasmacytoma, 8 AL amyloidosis and 126 classic MM) and 16 control subjects using a commercial ELISA kit. In 43 newly diagnosed patients sBCMA levels were measured at multiple time points during treatment and compared to conventional IMWG response and progression free survival (PFS). Results: sBCMA levels among control subjects were significantly lower than among newly diagnosed or relapsed MM patients [21.7 (18.5-24.9) ng/ml vs. 829.1 (665.3-992.7) and 543.2 (451.6-634.9) ng/ml respectively]. Significant correlations were found between sBCMA and the degree of bone marrow plasma cell infiltration. Out of the 37 newly diagnosed patients who have reached partial response or better per IMWG criteria, 33 (89%) have had at least a 50% drop in sBCMA level by therapy week 4. Cohorts of newly diagnosed patients made similarly to IMWG response criteria – achieving a 50% or 90% drop in sBCMA levels compared to the level at diagnosis – had statistically significant differences in PFS (Figure 1). Summary/Conclusion: Our results confirmed that sBCMA levels are prognostic at important decision points in myeloma. Importantly, the percentage of BCMA change is predictive for PFS. This highlights the great potential use of sBCMA in oligo- and non-secretory myeloma.Keywords: Multiple myeloma
PB2487: SECONDARY HLH: RETROSPECTIVE DATA FROM TERTIARY CARE CENTRE.
Authors: Nilesh Wasekar, Shivani Joshi
Journal: HemaSphere (2023)

Topic: 23. Hematopoiesis, stem cells and microenvironment Background: Hemophagocytic Lymphohistiocytosis (HLH) is an often-fatal syndrome of uncontrolled immune activation caused by dysregulation of macrophages and lymphocytes resulting in increased levels of inflammatory cytokines and significant tissue damage. Here we present 20 cases of adult secondary HLH that were identified and treated at Nashik Hematology Services in a period of two years. Aims: To highlight the presentation and etiology of secondary HLH and the importance of prompt recognition and early treatment. Methods: Between Jan 2020 to January 2023 20 adult patients >18 years old were identified at Nashik Hematology Services that met criteria and were treated for adult secondary HLH. All 20 patients had 5 out of 8 of the following findings established in the HLH-2004 guidelines: fever ≥ 38.5 C°, splenomegaly, cytopenias affecting 2 of 3 lineages in the peripheral blood (hemoglobin <9 g/dL, platelets <100x103/mL, neutrophils <1x103/mL), hypertriglyceridemia (fasting >265 mg/dL) and/or hypofibrinogenemia (<150 mg/dL), hemophagocytosis in the bone marrow, spleen, lymph nodes or liver, low or absent NK cell activity, ferritin >500 ng/mL and elevated sIL2R. Patients were treated with immunotherapy mainly dexamethasone and Ivig based on the previous experience of treatment of secondary HLH, as well as treatment targeted at an underlying malignancy or infection. Patient demographics and clinical characteristics, underlying etiology of secondary HLH and treatment modalities were compared. Results: All 20 patients included in this study met criteria for secondary HLH as defined above. The median age at diagnosis was 30 (range 22-68 years). Five out of 20 (25%) patients had Dengue fever. The median ferritin at diagnosis was 3000 ng/mL (range 1,000-10,500 ng/mL). Patient clinical manifestations are summarized in Table 2. All patients presented with fevers, elevated ferritin, cytopenias and splenomegaly and 15/20(75%) patients had evidence of hemophagocytosis in the bone marrow. 19 patients (95%) had secondary HLH due to an infection and 1(5%) had primary HLH. All patients received Dexamethasone and treatment for underling infection. At the time of this report, 5 patients had died (25 %), 14 were alive and 1 patient was lost to follow up. Summary/Conclusion: Diagnosis of the adult secondary HLH remains a challenge due to overlap in symptoms and lack of specificity of laboratory findings. Treatment is often postponed due to delayed recognition of this deadly disease. A high degree of medical suspicion in the appropriate clinical context should prompt early treatment with an Dexamethazone and Etoposide-containing regimen. Prospective studies are warranted in this area.Keywords: Hemophagocytic Lymphohistiocytosis (HLH)
PB2543: CIRCULATING MICROVESICLES AS POTENTIAL CELLULAR BIOMARKERS FOR THROMBOTIC RISKS IN BETA THALASSEMIA
Authors: Mariem Chebbi, Khouloud Khalfaoui, Sihem Saidani, Ines Safra, Mbarka Barmate, Dorra Chaouechi, Monia Benkhaled, Monia Ouederni, Fethi Mellouli, Samia Menif, Imen Moumni
Journal: HemaSphere (2023)

Topic: 27. Thalassemias Background: Microvesicles (MVs) are submicrometric fragments (0.1 to 1 μm) that shed from the remodeling of the plasma membrane of cells undergoing apoptosis or activation. They express high levels of phosphatidylserine on their outer leaflet together with other surface markers from their cell of origin. MVs were first neglected and described as circulating platelet “dust” in plasma, however, recently, increasing focus has been turned to MVs, for their pathogenic roles have been studied in various diseases such as beta thalassemia. It is an inherited blood disorder caused by reduced or absent synthesis of the beta globin chains of hemoglobin. The imbalance between the alpha globin and non-alpha globin chains causes a disruption of the membrane phospholipids in red blood cells which facilitates membrane blebbing and the release of pro-coagulant MVs. This increases the risk of thrombotic complications observed in patients. Aims: Our main focus is the research of circulating microvesicles as potential cellular biomarkers for thrombotic risks in beta thalassemia. Methods: Clinically diagnosed polytransfused beta thalassemia major patients from the National Bone Marrow Transplant Center in Tunis and healthy donors were sampled. Flow cytometry was carried out in order to quantify the apoptotic MVs originating from red blood cells, platelets and endothelial cells. Results: Our results demonstrated an increase in the apoptotic MVs, suggesting excessive apoptosis rate in patients compared to healthy donors. We also observed that MVs originating from endothelial cells and red blood cells were elevated in patients, which suggests their potential contribution to thrombotic risk. Summary/Conclusion: Our results suggest that MVs could serve as potential cellular biomarkers for thrombotic complications in beta-thalassemia. Keywords: Hemoglobinopathy, Microvesicles, Thrombosis
PB1958: IBRUTINIB AS THIRD LINE THERAPY IN CLL PATIENTS, A 5-YEAR STUDY IN ALBANIA
Authors: Shaqir Qerama, Liri Seraj, Jora Kociaj, Lorena Buhaljoti
Journal: HemaSphere (2023)

Topic: 6. Chronic lymphocytic leukemia and related disorders - Clinical Background: Chronic lymphocytic leukemia (CLL) is the most prevalent adult leukemia in Albania with new patients diagnosed almost every day. CLL mainly effects older patients and presents a variable disease course. The diagnosis of CLL requires the presence of more than 5 × 109/L B lymphocytes in the peripheral blood, sustained at least 3 months. CLL cells coexpress the surface antigen CD5 together with the B-cell antigens CD19, CD20 and CD23. Only patients with advanced, active and symptomatic disease need therapy. Ibrutinib is a transformative therapy for high-risk and relapsed refractory chronic lymphocytic leukaemia (CLL) patients. Aims: This study aims to find the clinical outcomes of patients treated in Albania since 2018 with Ibrutinib as third line therapy, the long-term efficacy, side effects and overall benefits for the patients treated. The research took place from July 2018 to December 2022. Methods: This study was carried out with data collected from the Statistic Department of the University Medical Center “Mother Teresa” Tirana and Hematology department. 44 consecutive patients diagnosed with CLL through a preliminary clinical and cyto-morphologic examination were included in the study. Bone marrow aspirations and peripheral blood EDTA samples were taken. The fluorescent stained monoclonal antibodies used were as following: CD5, CD20, CD19, CD23, CD10, CD34, CD56, CD3, CD4, CD8, CD38, KAPPA, LAMBDA. Data were analysed using SPSS. 25 IMB Results: The median age of the patients in this study resulted 65 years (interval of reference 42-75 years). 11 (25%) of them were females and 33 (75%) males. Our data confirm that single agent ibrutinib at 420mg daily (140mg x3) achieved good overall response rate (ORRs) of 66 %, and a median overall survival of 105.17 ± 7,6 months (interval 90 – 120 months) After 1 year of treatment with ibrutinib, all patients but one had an increase in red blood cells. Before treatment with ibrutinib only 72% of our patients were classified as stage C Binnet and after treatment their platelets increased by 88%. 41% of the patients had before treatment more than 50x103 white blood cells in peripheral blood, which was reduced to 11% after treatment with ibrutinib. Summary/Conclusion: Using Ibrutinib as third line therapy in resource poor country such as Albania has proven to positively affect the overall survival rate and significantly improve the complete blood count parameters.Keywords: Survival, ibrutinib, B-CLL
P1652: SAFETY OF DOACS IN PATIENTS WITH CHRONIC KIDNEY DISEASE: A SYSTEMATIC REVIEW AND META-ANALYSIS
Authors: Lucy Zhao, Daniel Tham, Wendy Yu, Jayhan Kherani, Allen LI, Pei Ye LI, Ali Eshaghpour, Mark Crowther
Journal: HemaSphere (2023)

Topic: 34. Thrombosis and vascular biology - Biology & Translational Research Background: Direct oral anticoagulants (DOACs) are now the standard of care for management of venous thromboembolism (VTE) and stroke prophylaxis in atrial fibrillation. Patients with chronic kidney disease (CKD) are at increased risk of VTE and atrial fibrillation. As DOACs are partially renally cleared and patients with CrCl<25-30 were excluded from large trials, there remains question of the safety of DOACs in patients with CKD. Aims: To synthesize primary evidence on the safety profile of DOACs in patients with CKD. Methods: We conducted a search of MEDLINE and Embase from database inception to January 2023. We included randomized controlled trials (RCTs) and cohort studies of any DOACs compared to vitamin K antagonists for prevention of thrombosis in patients with CKD. Two reviewers screened and extracted data at title/abstract and full-text levels individually and in duplicate. Baseline characteristics, anticoagulation regimen, and CKD stage were extracted. Primary outcome was ISTH major bleeding stratified by CKD stage. Data were meta-analyzed using random-effects model and presented as odds ratios with corresponding 95% confidence intervals. Results: Of the 2772 articles screened, 33 cohort studies and 7 RCTs comprised of 283690 patients were included. There was a non-significant reduction of major bleeding that favored DOACs for stage 3 CKD (OR=0.78 [0.59, 1.05]), stage 4 CKD (OR=0.82 [0.52, 1.25]), and stage 5 CKD (OR= 0.84 [0.58, 1.21]). Summary/Conclusion: In this preliminary analysis comparing DOACs to VKAs, DOACs were associated with a statistically non-significant reduction in major bleeding across stage 3-5 CKD. Further analyses are in process which will stratify by DOAC agent, duration, dosage, renal replacement therapy, and include secondary efficacy outcomes. Figure 1. DOAC vs Vitamin K Antagonist for the Risk of Major Bleeding Stratified by CKD StageKeywords: Venous thromboembolism, Oral anticoagulant, Chronic renal failure